• Announced the submission of a marketing application
  • to the European Medicines Agency (EMA) for a gene therapy.
  • Read more.

Latest news


The 30 members of the Telethon Scientific Committee on June 20-21 gave their final assessment of 160 projects that have passed...


New hope of a cure for genetic mitochondrial diseases, which in children can cause slowing or stopping of growth, progressive encephalopathy, severe muscle disorders, cardiomyopathies.